- Application Submitted for the Treatment of Partial-Onset Seizures and Migraine
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Eton Pharmaceuticals Submits New Drug Application to the FDA for Topiramate Oral Solution (ET-101)
WATERTOWN, Mass., Oct. 06, 2020 (GLOBE NEWSWIRE) -- C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a biopharmaceutical company pioneering a new class of small-molecule drugs that selectively destroy disease-causing proteins through degradation, today announced the closing of its initial public offering of 11.04 million shares of common stock, including the full exercise of the underwriters’ option to purchase up to 1.44 million additional shares of common stock, at a public offering price of $19.00 per share. The aggregate gross proceeds to C4T from the offering were $209.76 million, before deducting underwriting discounts and commissions and other offering expenses. All of the shares in the offering were offered by C4T.
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C4 Therapeutics Announces Closing of Initial Public Offering and Full Exercise of the Underwriters’ Option to Purchase Additional Shares
GAITHERSBURG, Md., Oct. 13, 2020 (GLOBE NEWSWIRE) -- Novavax, Inc. (Nasdaq: NVAX) Novavax, Inc. (Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today announced the formation of a leadership team to advance NanoFlu to regulatory licensure and the promotion of Russell (Rip) Wilson, J.D./M.B.A., to Executive Vice President and the newly-created role of NanoFlu™ General Manager. Mr. Wilson will focus exclusively on leading efforts to advance NanoFlu, the company’s influenza vaccine candidate, through global licensure, as well as the exploration of a combined influenza/COVID-19 vaccine that could be used in a post-pandemic setting. Novavax announced results of its successful NanoFlu pivotal Phase 3 clinical trial earlier this year and intends to seek regulatory approval from the U.S. Food and Drug Administration (FDA) under the accelerated approval pathway previously granted to the company.
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Novavax Appoints Leadership Team to Advance NanoFlu through Regulatory Licensure
CRANBURY, N.J., Oct. 13, 2020 (GLOBE NEWSWIRE) -- PMV Pharmaceuticals, Inc., (Nasdaq: PMVP) a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53 mutations, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead product candidate PC14586, for the treatment of cancer patients with locally advanced or metastatic solid tumors that have a p53 Y220C mutation. There are currently no FDA-approved medicines that target the p53 Y220C mutation.
WARMINSTER, Pa., Oct. 13, 2020 (GLOBE NEWSWIRE) -- Arbutus Biopharma Corporation (Nasdaq: ABUS), a clinical-stage biopharmaceutical company primarily focused on developing a cure for people with chronic hepatitis B virus (HBV) infection as well as therapies to treat coronaviruses (including COVID-19), today announced that the Company will participate in a virtual fireside chat at the H.C. Wainwright HBV Mini-Conference on Tuesday, October 20, 2020 at 10:00 am ET.
Third quarter 2020 Revenue expected to exceed $1 million, representing an increase of greater than 35% sequentially and greater than 50% over Q3 2019
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electroCore Provides Business Update and Select Financial Guidance
WINNIPEG, Manitoba, Oct. 13, 2020 (GLOBE NEWSWIRE) -- Kane Biotech Inc. (TSX-V:KNE; OTCQB:KNBIF) (“Kane Biotech”) today announced that Kevin Cole will assume the role of President and CEO of STEM Animal Health Inc. (“STEM”), a subsidiary created from the recently formed joint venture with UK-based international veterinary products company, Animalcare Group PLC (AIM:ANCR) (“Animalcare”).
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STEM Animal Health Appoints New President and CEO
In a recent clinical trial for an immune cell therapy for lymphoma, 62% of patients experienced complete cancer clearance in spite of the fact that some of them were on their 5th line of treatment. Stem cell therapies have the potential to enact more of these paradigm-shifting treatments. Proposition 14 will continue to advance these therapies and bring them to full development as available cures.
The vision of stem cell therapy is that a physician can just as easily grab an IV bag full of therapeutic cells as they might draw a drug into a syringe. Conceived through Proposition 71 in 2004, the California Institute for Regenerative Medicine (CIRM) serves as a vehicle to support all aspects of stem cell research. Medical progress requires not just well-designed clinical studies but also a well-trained workforce, educated at the intersection of stem cell biology, engineering, and chemistry.
Since 2008, CIRM has supported the training of nearly 1300 Community College and California State University students for the emerging field of Regenerative Medicine through the Bridges to Stem Cell Research and Therapy Program. The Bridges Training Program has functioned as a pathway for first-generation and underrepresented students from Humboldt to San Diego, to all of the biomedical sectors startup and cell therapy companies, academic research institutes, graduate and medical school, and more. Exposure to hands-on labs, advanced seminar discussions, and a required paid internship fully prepares these students for entering the stem cell workforce. Over 80% of Bridges alumni have either advanced to graduate school or joined the biomedical workforce in industry or academic institutions. These Programs bring a greater return than the initial cost of training.
SPONSORED
Consider Vahid Hamzeinejad, a bright high school student, headed to UC Berkeley to begin his college career. Enter the Great Recession; Vahid found himself back at home, working non-stop to help keep his parents restaurant afloat. Not giving up on his commitment to an education, he enrolled at the College of the Canyons. After completing an Associates degree, Vahid transferred to Cal Poly, hoping to join the Bridges Program. After receiving the Bridges core training, Vahid started his internship at ViaCyte, where he continues to work today, as a critical member of the team supporting ViaCytes clinical development of a functional cure for diabetes. The nearly $30 billion that California currently spends on diabetes treatments could be significantly reduced, in no small part due to the efforts of a student that cost taxpayers $36,000 to educate. That is before considering the benefit to patients quality of life that would occur by replacing insulin pumps, glucose monitors, and constant vigilance with a stem-cell-derived tissue that regulates blood sugar levels biologically making and secreting its own insulin.
Passing Proposition 14 will enable this and other unparalleled treatments for diabetes, heart disease, cancer, and neurological disorders.
Signed,
Robert Kam and the CIRM Bridges Program
SPONSORED
Features
Oct 16, 2020
Karen Wils photo Shown at right is Karen Wils, the Chemo Beagler or the COVID Beagler.
ESCANABA The year 2020 has put me and most everybody else in a very unusual position.
We all feel a little trapped and uneasy thrown into a situation we have never faced before.
I always kind of thought I would need to have a stem cell transplant someday after I was diagnosed with multiple myeloma in 2008.
I often referred to this blood cancer as my pesky woodtick. Fortunately, I have been blessed with good control of the disease until this year.
Leaving home, going to the Mayo Clinic and going through transplant is enough excitement for one person, but to be doing that while COVID-19 plagues our country is almost too much.
To say the last few months have been an interesting journey would be putting it mildly.
As the summer drew to a close, many changes were closing in on me, too. My son went off to a new job, and my daughter left for college. The very next week, my husband and I left for Minnesota for an unknown amount of weeks for my stem cell transplant.
For three-and-a-half weeks we were at Mayo. My husband was my excellent care giver through the strong chemo, and the transplant of my own stem cells and the long road to recovery.
Doing all of this during a pandemic is really interesting. The Mayo Clinic takes COVID-19 very seriously. So with much testing, temperature taking, sanitizing and constant mask wearing, we maneuvered through the treatment.
Social distancing when you are staying at the Gift of Life Transplant House and so far from home is very difficult. Phone calls, Zoom and Facebook became my life link to the kids, family and friends.
After going through some pretty weak days, finally my new immune system was strong enough for me to go home.
Oh, the great joy of heading down those last miles from Menominee to Escanaba back into the land of trees and water!
Sleeping in my own bed, seeing my pets, and being able to stand in my own garden felt so wonderful, but the house was so quiet without the kids.
The COVID-19 cautions had to continue, too.
I wear my mask and feel a little bit trapped, like everybody else these days. I pray that I am making all healthy decisions when I encourage my children to go to church or my husband to go to the grocery store.
The COVID-19 virus threatens big things and little things like family traditions that have to be rethought. With great sadness, I postponed until next year our annual camp gathering/harvest festival. So many folks have been through canceled or postponed parties, birthdays, anniversaries and funerals.
As hard and as disappointing as this is, the important things remain. The trees still are changing to awesome autumn colors. The harvest season is in full swing. The wild animals are still thriving outside our windows, and our families are together even if we cant do everything we want to do right now.
As I get stronger every day, I thank God. I pray for a cure for COVID-19. Yoopers are tough and our traditions will thrive long after this pandemic!
Karen (Rose) Wils is a lifelong north Escanaba resident. Her folksy columns appear weekly in Lifestyles.
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COVID-19 and the trials of treatment | News, Sports, Jobs - Escanaba Daily Press
Details Category: DNA RNA and Cells Published on Friday, 16 October 2020 14:20 Hits: 121
First therapy recommended for full marketing authorization in the EU for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants
One-time treatment with Libmeldy has been shown to preserve cognitive and motor function in most patients
Libmeldy is backed by data across 35 patients with follow-up of up to 8 years post-treatment, demonstrating the potential durability of HSC gene therapy
BOSTON, MA, USA and LONDON, UK I October 16, 2020 I Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy (cryopreserved autologous CD34+ cells encoding the arylsulfatase-A, or ARSA, gene), an investigational gene therapy for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.
The CHMPs positive opinion will now be reviewed by theEuropean Commission(EC), which has the authority to grant marketing authorization for Libmeldy in theEuropean Union(EU). A final decision by the EC for Libmeldy is anticipated before the end of 2020. If approved, Libmeldy would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.
MLD is a very rare, severe genetic condition caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them. A majority of these patients pass away in childhood, with palliative care often as their only option.
Todays positive CHMP opinion for marketing authorization of Libmeldy is a remarkable achievement that we share with the MLD community, as it brings us closer to delivering a one-time, potentially transformative therapy for eligible children suffering from this devastating disease, said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. Data from the Libmeldy clinical program have demonstrated the potential for long-term positive effects on cognitive development and maintenance of motor function, translating to individual preservation of motor milestones such as the ability to sit, stand and/or walk without support, as well as attainment of cognitive skills like social interactions and school attendance, at ages at which untreated patients show severe motor and cognitive impairments.
Libmeldy is designed as a one-time gene therapy, developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, in which the patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying genetic condition with a single treatment.
This is an important milestone toward making the availability of HSC gene therapy a reality for more patients, and it also is extremely rewarding for our multi-disciplinary team at SR-Tiget who has worked relentlessly along this 15-year journey to move the seminal proof of principle studies to the first in-human testing of this therapy, said SR-Tiget director Luigi Naldini, M.D, Ph.D. The robust and durable clinical benefits observed in early-onset MLD patients who received HSC gene therapy are compelling, especially when compared to the natural history of the disease. These results also further illustrate our view that the HSC gene therapy approach has the potential to deliver transformative effects in other storage diseases as well, especially when the cells are designed to overexpress the functional enzyme and provide an enhanced supply of it to the affected tissues.
As a parent, watching your child start down a seemingly normal developmental path only to suddenly and rapidly lose some or all of his or her abilities is heart-wrenching, and the agony is even more acute knowing no approved therapies currently exist for MLD, said Georgina Morton, Chair of ArchAngel MLD Trust. Todays decision to advance Libmeldy to the final EC approval stage represents a huge step forward for the parents of these young children and for all of us in the MLD community.
We are extremely appreciative of the EMAs expedited and thorough review of Libmeldys marketing authorization application, considering the severity of MLD coupled with the limited treatment options available today for young patients, said Anne Dupraz, chief regulatory officer, Orchard Therapeutics. The Agencys collaboration on this assessment is a testament to their broader public health commitment to ensure timely evaluation of new medicines for diseases where a pressing unmet need exists.
Data Supporting the Clinical Profile of Libmeldy
The positive CHMP opinion is supported by clinical studies of Libmeldy in both pre- and early- symptomatic, early-onset MLD patients. Early-onset MLD encompasses the disease variants traditionally referred to as late infantile (LI) and early juvenile (EJ).
Clinical efficacy was based on the integrated analysis of results from 29 patients with early-onset MLD who were all treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation:
Clinical safety was evaluated in 35 patients with early-onset MLD:
Co-primary endpoints The co-primary endpoints of the integrated efficacy analysis were Gross Motor Function Measure (GMFM) total score and ARSA activity, both evaluated at 2 years post-treatment. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.
Pre-symptomatic LI and EJ patients treated with Libmeldy experienced significantly less deterioration in motor function at 2 years and 3 years post-treatment, as measured by GMFM total score, compared to age and disease subtype-matched untreated patients (p0.008). The mean difference between treated pre-symptomatic LI patients and age-matched untreated LI patients was 71.0% at year 2 and 79.8% at year 3. Similarly, the mean difference between treated pre-symptomatic EJ patients and age-matched untreated EJ patients was 52.4% at year 2 and 74.9% at year 3. Although not statistically significant, a clear difference in GMFM total score was also noted between treated early-symptomatic EJ patients and age-matched untreated EJ patients (28.7% at year 2; p=0.350 and 43.9% at year 3; p=0.054).
A statistically significant increase in ARSA activity in peripheral blood mononuclear cells was observed at 2 years post-treatment compared to pre-treatment in both pre-symptomatic patients (20.0-fold increase; p<0.001) and early-symptomatic patients (4.2-fold increase; p=0.004).
At the time of the integrated data analysis, all treated LI patients were alive with a follow-up post-treatment up to 7.5 years and 10 out of 13 treated EJ patients were alive with a follow-up post-treatment of up to 6.5 years. No treatment-related mortality has been reported in patients treated with Libmeldy.
Key secondary endpoints For EJ patients who were early-symptomatic when treated with Libmeldy, meaningful effects on motor development were demonstrated when these patients were treated before entering the rapidly progressive phase of the disease (IQ85 and Gross Motor Function Classification (GMFC)1). By 4 years post-disease onset, an estimated 62.5% of treated, early-symptomatic EJ MLD patients survived and maintained locomotion and ability to sit without support compared with 26.3% of untreated early-symptomatic EJ MLD patients, representing a delay in disease progression following treatment with Libmeldy.
A secondary efficacy endpoint that measured cognitive and language abilities as quantified by Intelligence Quotient/Development Quotient (IQ/DQ) found:
Clinical safety Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in 5 out of 35 patients. Antibody titers in all 5 patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. Treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.
About MLD and Investigational Libmeldy
Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in thearylsulfatase-A(ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. Currently, there are no approved treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), formerly OTL-200, is being studied for the treatment of MLD in certain patients. Libmeldy was acquired from GSK inApril 2018and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their jointSan Raffaele-Telethon Institute for Gene TherapyinMilan, initiated in 2010.
About Orchard
Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.
Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.
1Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669
SOURCE: Orchard Therapeutics
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Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) | DNA RNA and...